U.S. FDA accepts supplemental New Drug Application for OFEV® (nintedanib) for children and adolescents aged 6-17 years old with fibrosing interstitial lung disease

• The supplemental New Drug Application is based on results from the InPedILD phase III trial, which assessed dose-exposure and safety in pediatric patients aged 6-17
• If approved, OFEV would be the first and only approved treatment for these patients, helping to address an unmet need

RIDGEFIELD, Conn. and INGELHEIM, Germany, July 25, 2023 /PRNewswire/ -- Boehringer Ingelheim announced today that the U.S. Food and Drug Administration (FDA) accepted its supplemental New Drug Application (sNDA) for OFEV (nintedanib), investigating a potential treatment for children and adolescents between 6 to 17 years old with fibrosing interstitial lung disease (ILD).

"With a long and complicated journey to diagnosis and no approved therapies, childhood interstitial lung diseases can place a significant burden on patients and their families," said Prof. Robin Deterding, M.D., Director of the Breathing Institute, Children's Hospital Colorado. "If approved, OFEV would be the first and only approved treatment for children and adolescents aged 6-17 years old with fibrosing interstitial lung disease, representing an important step forward for these patients."

The sNDA is based on results from the InPedILD phase III trial, which evaluated dose-exposure and safety of OFEV on top of standard of care in children and adolescents aged 6-17 years old with clinically significant fibrosing ILD. In children and adolescents with fibrosing ILD, a weight-based dosing regimen resulted in exposure of nintedanib similar to adults and with a similar safety profile. The most common adverse event associated with nintedanib compared to placebo in the InPedILD trial was diarrhea. The complete results were published in the European Respiratory Journal (ERJ) and presented at the European Respiratory Society (ERS) International Congress in September 2022.

"We remain committed to building on our heritage in respiratory innovation with an aim to provide life-changing treatments for patients living with pulmonary fibrosis, including those living with childhood interstitial lung disease," said Craig Conoscenti, M.D., FCCP, ATSF, Executive Director/Therapeutic Area Head, Respiratory IPF/ILD, Clinical Development and Medical Affairs, Boehringer Ingelheim. "We are pleased that the FDA recognizes the unmet need for this patient population, and we look forward to working with the FDA to potentially bring forward the first approved option as quickly as possible." 

About childhood interstitial lung disease 
Childhood interstitial lung disease (chILD) is a rare condition that includes more than 200 disorders. The reported incidence of chILD ranges from 1.6 to 46 per million children, but its exact prevalence is unknown and pulmonary fibrosis within chILD is even less frequent, with no known global prevalence estimates. chILD is associated with significant mortality and morbidity. There are no established diagnostic criteria and few management guidelines.

About the InPedILD trial
InPedILD (NCT04093024) is an exploratory phase III double-blind, randomized, placebo-controlled trial assessing dose exposure and safety of nintedanib on top of standard of care for 24 weeks, followed by open-label treatment with nintedanib of variable duration in children and adolescents aged 6-17 years with clinically significant fibrosing ILD. It is one of the first randomized controlled clinical trials focused on childhood interstitial lung diseases. The co-primary endpoints were dose-exposure at week 2 of nintedanib treatment (i.e. at week 2 in patients randomized to receive nintedanib and at week 26 in patients randomized to receive placebo for 24 weeks followed by nintedanib) and the proportion of patients with treatment-emergent adverse events at week 24. Of 87 patients screened, 39 patients (12 patients aged 6?11 years and 27 patients aged 12?17 years) were randomized and treated (26 patients with nintedanib and 13 patients with placebo).  

What is OFEV?

• OFEV is a prescription medicine used:
• to treat adults with a lung disease called idiopathic pulmonary fibrosis (IPF).
• to treat adults with a long lasting (chronic) interstitial lung disease in which lung fibrosis continues to worsen (progress).
• to slow the rate of decline in lung function in adults with systemic sclerosis-associated interstitial lung disease (SSc-ILD) (also known as scleroderma-associated ILD).
• It is not known if OFEV is safe and effective in children.

Important Safety Information 

What is the most important information I should know about OFEV? 

OFEV can cause harm, birth defects, or death to an unborn baby. Women should not become pregnant while taking OFEV. Women who are able to become pregnant should have a pregnancy test before starting treatment and should use highly effective birth control at the start of treatment, during treatment, and for at least 3 months after your last dose. Talk with your doctor about what birth control method is right for you during this time. Birth control pills may not work as well in women having vomiting, diarrhea, or other problems reducing the drug absorption. If you have any of these problems, talk with your doctor about what highly effective birth control method is right for you. If you become pregnant or think you are pregnant while taking OFEV, tell your doctor right away. 

What should I tell my doctor before using OFEV? 

Before you take OFEV, tell your doctor about all of your medical conditions, including if you have: 

• liver problems. 
• heart problems. 
• a history of blood clots. 
• a bleeding problem or a family history of a bleeding problem. 
• had recent surgery in your stomach (abdominal) area. 

Tell your doctor if you: 

• are pregnant or plan to become pregnant. 
• are breastfeeding or plan to breastfeed. It is not known if OFEV passes into your breast milk. 

You should not breastfeed while taking OFEV. 

• are a smoker. You should stop smoking prior to taking OFEV and avoid smoking during treatment. 

Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements such as St. John's wort. 

What are the possible side effects of OFEV? 

OFEV may cause serious side effects. 

TELL YOUR DOCTOR RIGHT AWAY if you are experiencing any side effects, including: 

• Liver problems. Unexplained symptoms may include yellowing of your skin or the white part of your eyes (jaundice), dark or brown (tea-colored) urine, pain on the upper right side of your stomach area (abdomen), bleeding or bruising more easily than normal, feeling tired, or loss of appetite. Your doctor will do blood tests to check how well your liver is working before starting and during your treatment with OFEV.
• Diarrhea, nausea, and vomiting. Your doctor may recommend that you drink fluids or take medicine to treat these side effects. Tell your doctor if you have these symptoms, if they do not go away, or get worse, and if you are taking over-the-counter laxatives, stool softeners, and other medicines or dietary supplements. 
• Heart attack. Symptoms of a heart problem may include chest pain or pressure, pain in your arms, back, neck, or jaw, or shortness of breath. 
• Stroke. Symptoms of a stroke may include numbness or weakness on one side of your body, trouble talking, headache, or dizziness. 
• Bleeding problems. OFEV may increase your chances of having bleeding problems. Tell your doctor if you have unusual bleeding, bruising, wounds that do not heal, and/or if you are taking a blood thinner, including prescription blood thinners and over-the-counter aspirin. 
• Tear in your stomach or intestinal wall (perforation). OFEV may increase your chances of having a tear in your stomach or intestinal wall. Tell your doctor if you have pain or swelling in your stomach area. 
• Increased protein in your urine (proteinuria). OFEV may increase your chances of having protein in your urine. Tell your doctor if you have any signs and symptoms of protein in the urine such as foamy urine, swelling, including in your hands, arms, legs, or feet, or sudden weight gain. 

The most common side effects of OFEV are diarrhea, nausea, stomach pain, vomiting, liver problems, decreased appetite, headache, weight loss, and high blood pressure. 

These are not all the possible side effects of OFEV. For more information, ask your doctor or pharmacist. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. 

Please see full Prescribing Information, including Patient Information.

CL-OF-100056 01.18.2022

About Boehringer Ingelheim
Boehringer Ingelheim is working on breakthrough therapies that improve the lives of humans and animals. As a leading research-driven biopharmaceutical company, the company creates value through innovation in areas of high unmet medical need. Founded in 1885 and family-owned ever since, Boehringer Ingelheim takes a long-term perspective. Around 52,000 employees serve more than 130 markets in the three business areas, Human Pharma, Animal Health, and Biopharmaceutical Contract Manufacturing.
Learn more at www.boehringer-ingelheim.us 

Media Contact
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Boehringer Ingelheim Pharmaceuticals, Inc.
Phone: +1 203-561-2829
Email: kerry.wycislo@boehringer-ingelheim.com

SOURCE Boehringer Ingelheim